Healing Parkinson’s patients with their own stem cells

Up to 1 million Americans have Parkinson’s, according to the Parkinson’s Disease Foundation. Because aging is the chief risk factor for the disease, the patient population is expected to increase as the baby boom generation gets older. Parkinson’s selectively kills brain cells that make the neurotransmitter dopamine, which enables movement. No one knows how it happens, or how to stop it. Researchers expect that transplanted dopamine-producing brain cells will eventually die, but perhaps not for 10 to 15 years.

The most visible symptoms of Parkinson’s include tremors, slowed movement, stooped posture and loss of balance, and trouble speaking. People sometimes walk with a shuffling gait, and they may experience severe and chronic pain. Patients’ faces can assume a mask-like expression. Drugs that provide dopamine or mimic its effects can partially relieve the symptoms, but they produce side effects such as uncontrolled movement. Also, their effectiveness decreases over time.

A groundbreaking stem cell treatment for Parkinson’s disease is getting close to moving from lab research in La Jolla to therapy for patients. The research, funded by the patients and their supporters, could also pioneer a new model for moving medical advances from the lab into the clinic.

Eight Parkinson’s patients have allied with scientists from The Scripps Research Institute and medical professionals from Scripps Clinic for the project, which involves creating new brain cells from other cells in their own bodies. Because of the unusual, personalized nature of the research, the patients are participating with scientists and doctors as equals, meeting regularly to review the progress.

The ambitious goal is to relieve the movement difficulties Parkinson’s causes by replacing the brain cells the disease destroys. In theory, it would restore near-normal movement for a decade or more, and the procedure could be repeated as needed.

Research is far enough along that scientists and health care professionals in the project are talking to regulators about beginning clinical trials, perhaps as soon as next year.

The replacement brain cells are now being grown in a lab at The Scripps Research Institute. Patches of skin the diameter of a pencil eraser were removed from the patients’ arms and turned into a new kind of stem cell that acts like embryonic stem cells. Called induced pluripotent stem cells, they were discovered in 2006, a feat honored by a Nobel Prize last year.

These IPS cells can become nearly any kind of cell in the body… Another potential advantage of IPS cells over embryonic stem cells is that they should be less prone to rejection by the patients’ immune systems, because the transplanted cells come from the individuals themselves.

Patient Cassandra Peters, 57, learned of the reality of Parkinson’s and the hope of a new treatment in a visit with Dr. Houser, her neurologist. “Interestingly, when I first had a conversation with her, when she definitively told me I had Parkinson’s, she said to me, quote, “You will have a stem cell procedure in your lifetime.” I took that ball and held it in my heart, thinking, this is going to be my ‘get out of jail free’ card. Not a day goes by when I don’t have an opportunity to share what I’m going through now and what the future might hold,” Peters said.

Ileana Slavin, a research associate in the lab of Jeanne Loring, and Suzanne Peterson, a staff scientist, discuss what it means for scientists to directly meet the people they’re trying to help. Diabetes researcher Matthias von Herrath of the La Jolla Institute for Allergy & Immunology said the work could help scientists developing stem cell therapies for diabetics,” von Herrath said. “And that’s going to open the door for these type of stem cells.”

Loring’s researchers are reaching the final stages of their part of the project. They have made induced pluripotent stem cells from all eight patients, and have turned those into the needed brain cells for two of them. The work continues for the other six.

Parkinson’s represents the “low-hanging fruit” of neurological diseases for stem cell therapy. We know what cell types are lost in Parkinson’s disease,” Bratt-Leal said in a March 8 meeting of the group. “We can make them from stem cells. And now we can make stem cells from adult tissues. The next logical step is to make these cells from people and put them back into them.”

“With IPS cells grown from the patient, rejection should be less of a worry”, Bratt-Leal said.

Now that the research side of the project has overcome its greatest hurdles, the focus is shifting to medicine, Loring said. The replacement brain cells will be grown in a clinical grade facility at the City of Hope in Los Angeles. As part of the transition to the medical side, Houser will provide expertise in setting up the clinical trial, assuming approval is granted by the U.S. Food and Drug Administration.

Beyond the potential benefit to the eight patients, the project may provide an answer to what Loring and other researchers call the “Valley of Death,” the period that halts promising research before it can become a medical treatment. Most scientific research is federally funded, but commercialization is left to the private sector. If companies don’t see a way to make money, they won’t pursue a therapy, even if it works. This problem is especially forbidding for treatments customized to individual patients. These don’t produce economies of scale, and hence are not attractive to pharmaceutical companies. Advocates of the customized Parkinson’s therapy said it will pay off in the long run. Patients will require less medical care, and find it easier to maintain their jobs.

LA JOLLA, Calif. & CAMBRIDGE, Mass. – (Business Wire) Fate Therapeutics, Inc. announced today that Dr. Rudolf Jaenisch, M.D., founding member of the Whitehead Institute for Biomedical Research and professor in the Department of Biology, Massachusetts Institute of Technology, has joined the Company’s internationally recognized team of scientific founders dedicated to understanding stem cell biology in human physiology and disease.

Fate Therapeutics is interrogating stem cell biology to develop therapeutics based on modulating cell fate and to enable a new drug discovery paradigm with the Company’s proprietary induced-pluripotent stem (iPS) cell technology. The Company’s first therapeutic candidate is scheduled to enter clinical trials in early 2009 in hematopoietic reconstitution. Fate Therapeutics is a private biotech company headquartered in La Jolla, CA.